This year, the Right to Try act was signed into federal law. This law allows patients to request the use of investigational drugs (without FDA involvement or state law). There was a current "compassionate use" framework, but no actual law. This law allows for drugs as early as Phase I to be used. Patients may be charged the direct cost of treatment, but the company cannot make a profit.
Personally I cannot think of any negative issues with this. This law is meant for those who have a life-threatening disease/condition. At this point they would exhaust all possible options, but if there are no current solutions, they should be able to choose to try a potentially life saving drug. It may seem like there may be ethical issues with this, but I believe the current set-up for the law alleviates those worries. Hope is something that these patients deserve, and though these drugs may not save them, at least they can give them some hope.
What is your opinion on this? Feel free to add more of your own research.
I have a very positive opinion on the Right to Try Law. I think that terminal patients should be allowed to try an experimental treatment despite the possible risk of death. It makes sense because cancer patients will die regardless of whether or not they get treatment. The Right to Try Act allows them one last chance to get cured. Even if it doesn't work and the drug treatment kills the patient, at least it would be known that the drug doesn't work and that every viable option to save the person's life was considered. Having hope that the treatment will work can also do some good as well. It's important that patients have some hope that their lives will be saved. Their conditions can potentially get worse if they have a negative attitude and mindset. This law applies to only patients who have a life-threatening condition so it makes sense that people who are dying should be allowed one last chance to save their own lives by choosing to go through with whatever treatment is proposed, despite the possibility of death. The Right to Try laws have been enacted in over 41 states so 9 states have not signed this into law. There is one particular drawback to this legislation, which is that pharmaceutical companies or drug manufacturers do not have an obligation to provide the drug therapies requested.
I believe the new Right to Try law is a good move for the most part for the obvious reasons. The best case-scenario is that the patient recovers with the help of an investigational treatment and that company attains validation for their product. Validation for drugs and medical treatments take an incredible amount of time and resources to ever reach human trials so having the ability to bypass those steps even for a few human trials is extremely beneficial, even if the patient does not recover. However, I do see how this may cause potential problems down the line. First off, it is important to understand that just because an individual takes a drug, does not mean only that individual is affected. We all naturally release and leave behind bodily fluids, dead skin, and hair. Not to mention we exchange our bodily fluids with others in various different ways every day. With that said, how can anyone be certain that the trace amounts of a particular drug, found in our bodily fluids, skin, and hair, will not have an impact on others? This is particularly important if the drug consists of nanoparticles. I'm sure this may be far-fetched but these are things that should be considered when exposing any drug outside of a controlled and contained environment.
Right to try law allows terminally ill patients to have access to drugs that are not yet approved by the FDA. If all other treatments have been attempted and resulted in failure and there is a high chance that the patient will soon die, it makes perfect sense for them to want to approach experimental drugs as a last chance at survival. At that point they have nothing to lose, yet something to gain in the case that the drugs do actually help to prolong their lives or in the most extreme case cure their disease. It is definitely worth it to give it a try. Also, I agree with @Dkonara921 that having a negative attitude can make matters worse. I think psychology plays a huge role on how people feel physically. Having hope that there is still something that they can try will have a positive effect.
I think this law is huge step forward on the clinical world. I can only imagine the difficulty that comes with finding willing participants for certain experiments. Dr. Simon mentioned some college buddies who would try these new drugs in order to make a quick buck, but perhaps did not realize the risk they put themselves in the process. For most, a monetary reward is the only thing enticing them to agree to the trials, as they may be in a desperate point in their lives and willing to do anything. For some trials though, financial compensation may not be enough for anyone. I know in the past before the FDA was really established and informed consent was law, doctors would perform experiments on any patients they could take advantage of such as people with mental illness, mental handicaps, or the homeless. This way if anything went wrong they may be able to avoid any consequences. The Right To Try law gives even the most risky drugs and devices a chance at making a medical breakthrough. I know if I was terminally ill and there was no progress after exhausting all approved options, I would be willing to try anything if it was a chance at a few more months or improved bodily function. Unfortunately some advances cannot be achieved without some casualties, but in sacrificing a few, many can be saved and this is one way to accomplish that legally and ethically.
I have conflicting opinions about the Right to Try Law. Clearly, if someone is seriously ill and all medical treatment options have been exhausted, and the doctors say that there is no FDA-approved medication that would help me, however, there’s a drug company that developed a treatment that might work, even with a 1% chance, it is reasonable that the patient’s will is honored and his wish is granted in that slight hope. On the other hand, this might be exploited by some companies, even if the current law allows only for charging the patient the direct cost of treatment, and not a profitable charge. I think that Big Pharma companies would find a way to make profits of these non-FDA approved drugs. How? One classical trick is to employ a US-written law and advertise that abroad and make the case that if it was that dangerous it would not have been allowed in the US, to begin with. With a slight overcharge on the drug transportation overseas, Big Pharma might push their agenda on foreign medical centers where bribery and corruption are common and all of a sudden, their non-FDA approved drug may end up being the first in line to that particular disease. Not to mention that if we allow non-FDA approved drugs to be consumed legally, that would leave the door wide open for that company to create a buzz in the drugs black market globally. On the contrary, if the FDA has a firm stand that a particular drug has no positive effect on the patient’s treatment, and might result in fatal effects, the company won’t have that strong of a case to advertise for their medication in question.
Right to Try seems to be a good step in terms of validating the drugs that companies make, assuming that it continues to use the "compassionate use" case in order to decide who gets these drugs. For patients that have no other approved option to turn to, potentially life saving drugs could be given to patients even if all known options have been exhausted. This also is a positive in the eyes of the drug maker as there can be early validation that their drug works (or not). Overall though, the decision of Right to Try is very risky for both parties. There is by no means any guarantee that the tried drug will even help; the drug may even make the situation worse for the patient. For the company as well, letting others try the drugs before it is cleared runs the risk of any failures blacklisting the company's name in the public's eyes, should anyone get worse or die. Should this be done, it should be, as it usually is, done in a way in which both parties are aware of the not only the positive possibilities, but the negative ones as well.
The right to try law seems to be a viable option for those who have no other hope. Critics argue that such experimental trials can harm the patient, but these drugs have already been approved for clinical trials. That means that other people are taking the same risks. I do however think that the doctors of the patient should be well versed on the nature of treatment and should be kept up to date with the results of the clinical trials. That was one of the concerns of the critics of this act. Although little may be known about the treatment the doctor should learn as much as they can about it.
Update to the topic:
The right to try law passed in May 2018 has only 2 patients as of June 2019 being accepted for experimental therapy.
Even I see no negative issues with the law and ideally, more people should have made use of the law.
The newspaper articles say that though the law has been made, the doctors are not aware of where the new drugs are so there needs to be a channel through which all the doctors, as well as the people, involved, know how to take part in the experiment.
Companies are also reluctant to do an experiment on the patient as if anything goes wrong the sponsor might want to back out.
Also, company reputation could also go down if the drug experiment doesn't go as per plan.
I think for the most part everyone is in agreement that the Right to Try act is good thing; it gives people who are close to losing hope a chance to try something risky but potentially life saving, and as a person's health declines, so does their risk.
The infrastructure of our medical system in the US has a little bit of work to do to actually make this accessible. Some hospitals are very advanced in their research and connections to research facilities that work on finding new treatments to diseases that don't currently have one that can help all patients or any at all. These hospitals are usually the ones to make the benefits of Right to Try more accessible because they have the reach to get a patient the experimental treatment they could be looking for.
While most hospitals have some kind of "reach", most can't service every kind of patient with ground breaking medical treatments. For example, some hospitals are known for being very good at cancer treatment and research, and so they can easily provide experimental cancer treatments faster than other hospitals could under the Right to Try act. However, that same hospital probably isn't the best around for various other kinds of diseases, so they won't be able to provide the same level of care and assurance as another one for other diseases. Therefore there needs to be some kind of network to easily connect patients willing to use the Right to Try with researchers and doctors who can provide the level of care to the patient and their experimental treatment.
I actually didn't know this law existed! At a glance it sounds like a good thing. For terminally ill patients this could provide a sliver of hope or a chance for them to contribute to science. Given my limited knowledge on the subject I searched and found an article on the pros and cons to this law, a quick summary listed below: http://jnm.snmjournals.org/content/59/10/1492.full
PROS:
1. Potential to extend life if the drug proves even slightly effective
2. More options for patients giving them more control of their access to experimental treatments
CONS:
1. Limited patient understanding; a false hope could result in massive disappointment; the drug could worsen the conditions
2. Financial consequences for patients: expenses are out of pocket
3. Safety Consequences: there is a high possibility of adverse consequences
As of January 2020, it has been reported that one patient with amyotrophic lateral sclerosis, or ALS, has received an investigational drug from BrainStorm Cell Therapeutics and seven patients have received an immunotherapy vaccine under evaluation for glioblastoma under the Right to Try Act. Now many terminally ill patients who are living with cancer consult their oncologists to request for Right to Try Act. But clinicians should not mislead them and instead educate patients about reasonable expectations for any experimental therapy and seek to identify and discuss equally promising options.
In most circumstances, I advocate the “Right to Care” act due to the simple fact that this may be the last resort for terminally ill patients. Rather than focusing on the pros of this act, I would like to focus on some of the cons since many of the responses in this discussion covered the benefits. For starters, there is often an issue with liability regarding adverse effects of treatment for physicians and manufacturers. Even though the general well-being of the patient is considered by physicians and manufacturers when providing experimental treatment, the safety of the drug if often unknown and can very well cause adverse effects. Additionally, actually understanding how treatment works can be difficult for the patient to understand due to the complexity of the scientific theory. This can lead a patient into accepting a treatment that they do not fully understand which may provide results that they did not anticipate. Aside from these two cons to the “Right to Try” act, patients who seek these treatments are often seeking their last resort on treatment and in many cases are willing to sacrifice their life for seeking overall treatment.
I think any ethical issues related to the Right to Try Act can be mitigated by clear communication between the physicians and the patients. The RTA specifies that the patient must be diagnosed with a terminal illness. I think participation in this program is a win-win scenario. The physicians have the opportunity to measure important clinical data. The patient gets a chance to improve their condition.
I agree with the Right to Try Act. Individuals should consent to test without the FDA approval or state law if they are in dire need and save their lives. The only part that I find that I'm torn between is that companies can charge them the total cost of the treatment if it has not been proven as a treatment. On the one hand, I would say the companies cannot charge them the total cost. Still, I also understand that the company may already have all of the patients they need for their study, and to include others would only drive up their testing cost, which would make sense for the patient to have to cover their treatment. But overall, I do agree with the Right to Try.